A group of scientists have created 10 disease-specific stem cell strains. They will make the cell lines available worldwide through a facility funded by the Harvard Stem Cell Institute.
The team developed these stem cell lines for Duchenne muscular dystrophy, Becker muscular dystrophy, juvenile-onset (type I) diabetes, Parkinson's disease, Huntington's disease, Down's syndrome, ADA severe combined immunodeficiency, Shwachman-Bodian-Diamond syndrome, Gaucher disease, and Lesch-Nyhan syndrome.
In many cases these new stem cell cultures will mimic human disease more reliably than animal models, according to Howard Hughes Medical Institute investigator George Q. Daley, who led the work. The technique will even enable the comparison of how the same disease varies among people, by generating disease-specific stem cell cultures from many individuals. The cells will also offer a proving ground for screening drugs to treat disease.
To produce the disease-specific stem cells, the researchers mixed cells from patients with the disorders with benign viruses to introduce certain previously discovered reprogramming factors into the cells.
Once the investigators isolated the disease-specific stem cells, they analyzed the genes and confirmed that the stem cells had the same disease-causing defects as the original donor cells. The researchers also made sure that the stem cells were pluripotent.
Scientists from Harvard Medical School, Massachusetts General Hospital, and the University of Washington were also involved in this study. Their results appear in an advanced online August 7 publication of Cell.